Drug Discovery World

NOVEMBER 7, 2022

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. That said, some people with rare genetic diseases remain without a molecular diagnosis after their genome is analysed. . “We

Genome 52

Genome Genomics Genetics Genetic Disease 52

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). Krystal expects Vyjuvek to be available by the third quarter of this year.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

Genetic Disease 85

Genetic Disease Protein RNA Doctors 85

pharmaphorum

AUGUST 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans. The three drugs required intravenous dosing once a week and work only in around a third of DMD patients.

Gene Therapy 100

Gene Therapy Gene Trials Genetics 100

The Pharma Data

DECEMBER 3, 2020

Antiepileptic Drugs / 7B. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome. Secondary and exploratory endpoints in the study will evaluate changes in other disease measures, including seizures and additional non-seizure comorbidities. 4, 2020 14:57 UTC. BEDFORD, Mass.–(

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

XTalks

OCTOBER 1, 2020

Xtalks spoke with WeiQi Lin, MD, PhD, executive vice president, research & development, and principal scientist at DURECT Corporation — a company focused on developing drugs and drug delivery methods in therapeutic areas with significant unmet need — about her transition from academia to industry. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52